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Below is a transcript from a Town Hall meeting to gather evidence of whether to continue Medicaid funding for renal stenting. We hear from Dr. Lance Dworkin, senior leader for the CORAL trial.

"So, in conclusion, randomized trials are expensive, they’re difficult to perform and they’re uncommon, enrollment is a major barrier. Coverage policies, I think, do affect enrollment by altering the chances that an unproven therapy will be provided outside of the study. Registries are not the same as randomized clinical trials and can substitute for them. Clinical practice guidelines based on observational danger - data I think may increase cost without improving patient outcomes and also discourage enrollment."



Amy Abernethy:

Okay? Thank you. Next, we’re going to move forward with Dr. Lance Dworkin, a Professor of Medicine and Vice Chairman of - for - of for Medicine for Research and Academic Affairs, as well as the Director of the Division of Kidney Research and Hypertension at the Alpert Medical School of Brown University in Providence. He has laboratory researched in kidney failure as well as is a clinical specialist in hypertension and is Study Chair and senior leader for the CORAL trial. This is a study in renal artery stenosis and this study is pertinent to us in today’s discussion. Lance Dworkin: Thank you. So I’m not an expert on health policy or funding for health policy. I’m an academic physician and a clinical trialist. And I wanted to describe to you the experience of leading a trial which is currently ongoing and which is struggling to enroll patients and some of our thoughts about why that might be. So, first of all, I think one of the points to make here is that it’s very, very difficult to do clinical trials and there is in fact a paucity of clinical trials, particularly randomized clinical trials that are being done. And I think this is a major issue for medicine. This is a slide recently published in one of the kidney journals looking at the percent of public studies that are randomized clinical trials in the literature. And the point is that it’s a very small percentage of all published studies. And in my area, which is nephrology, only about 2% of all published studies are randomized clinical trials. So I think all physicians agree that this is exactly the best kind of evidence of what we’re looking to do but somehow we’re failing to succeed in this area. So I just wanted to tell you a little bit about our study. And hopefully, that will shed some light on the question. And this is not a cancer trial, and it’s not even really a drug trial. So maybe, it’s a little bit different than what you’ve been hearing. We’re comparing an intervention, which is a renal revascularization or angioplasty and stenting, to medical therapy. And I think in designing and coming up with this clinical trial, there are a number of steps to this. So first of all, pick a clinical problem. And renal artery stenosis is a common clinical problem. It affects 1% to 5% of all hypertensives, about 70% or 7% of all elderly patients, many patients with vascular disease in other beds. Pick an area for which the best treatment is not known. In this particular area, there was an AHRQ review and I think - that’s, you know, maybe not a definitive statement on this area. It was actually done after the trial was launched. But they’ve reviewed all the pertinent evidence for or against revascularization in this disease and concluded that the available evidence did not clearly support one treatment approach over another. So here is an area where there’s clinical (unintelligible) where there’s really no evidence supporting one approach or another. So then, the next step is to ask a clinically relevant question and designing randomized clinical trials. I think one of the pitfalls is to look at surrogate endpoints rather than hard patient outcomes. And that’s something which we’ve tried not to do by looking at things like heart attacks and strokes in these patients rather than surrogate endpoints like blood pressure. So this is the way our trial is designed. It’s a randomized trial. Target enrollment is about 1100 with patients with about five years of follow-up. Our primary endpoint is a composite which includes some hard clinical outcomes like death, stroke, myocardial infarction. And the way the study is designed, it has about 90% power to look at the primary endpoint. And the way recruitment works is that we have a suspected patient, they sign consent, they undergo an imaging procedure that demonstrates the stenosis, and then they’re randomly assigned to either receive the intervention or not. And then everybody gets an intensive medical regimen. A big barrier to clinical research, of course, is funding. These trials are very expensive. I just mentioned this in passing. The cost of the CORAL trial to the NIH which is funding it is $30 million unless we run over budget, which we may. This is equivalent to 80 to 120 individual RO1 grants. So, a big disincentive to doing this kind of research is the cost involved. So this is, of course, the Achilles heel of most clinical research, is enrolling the subjects. Probably naively, we thought this would not be a problem in renal vascular disease, and we thought that all we had to do is find enough experienced centers because there were certainly enough procedures being done. And this is some data looking at the number of renal revascularizations being done. This is a booming business in the United States. The number of procedures has been increasing progressively. Most recently, the estimate is about 35,000 per year in the United States. And remember, we’re looking to accrue 1000 patients. There is a significant payment. A lot of these are paid for by Medicare. And the average cost, not including everything, probably is somewhere in the $2000 to $6000 per procedure. This just shows enrollment in the CORAL trial. The blue line was our projected enrollment when we applied for the grant; the yellow line is our revised; and the pink line is the actual enrollment. Between June 2006 and 2007, we enrolled 180 patients out of the 35,000 undergoing renal stenting in the United States. That’s .5% or 1 out of 200 of all procedures. And I just point to the fact that we had 456 reported screen failures. And these were patients who qualified by all other criteria, but failed to be enrolled either because of patient or physician preference. And I would suggest to you that this number is a very, very gross underestimation of the number of patients that didn’t enter the trial for this reason because oftentimes, patients or physicians wouldn’t even consider a patient for a trial because of their individual preference so that they wouldn’t be entered in the screening log. But if the procedure was not available except through the study, you’ll wonder how many of those patients would have - or physicians would have refused the entry for their patients. So how do - how we try to stimulate enrollment in this trial? We’ve made a lot of modifications to the protocols to make it easier. Patients get free medications. We’ve increased reimbursement for the centers. It’s interesting, I participated back in July in a (MedCAC) meeting right here in this room, which was examining the state of evidence and the impact of coverage policies on reimbursement for renal artery stenting. And some of the topics that were discussed at that meeting were it only covered procedures for patients that were enrolled in approved clinical trials. And I just want to talk about that and raise two issues related to that. And one has to do with registries versus randomized clinical trials. So a lot of times, I think when coverage is limited to approved clinical trials, that includes not only randomized clinical trials but also registries. And there are important differences. Registries collect information on patients undergoing a procedure or a therapy, but all of the patients entered in the registry actually receive that therapy. So they provide no useful information on the relative utility of the procedure or intervention versus no intervention. And I think they can sometimes be useful after a randomized trial’s demonstrated benefit to refine a clinical practice, but really shouldn’t precede clinical trials. And registries can undermine enrollment in clinical trials. Remember, all patients get the intervention - there’s no untreated group. So if you or your patient, if you’re the physician, believe in the procedure, then entry into the registry is preferred over entry into a randomized trial. There’s financial disincentives to randomization because if you’re getting reimbursed $6000 for the procedure and if you enter the patient in a registry, you get that 100% of the time. And if you enter the patient in a randomized trial, you get it 50% of the time. That’s a significant difference. And then there are also just, from the point of view of the conduct of clinical trials, big differences because oftentimes, the amount data being collected in a randomized trial is much greater than a registry. And so, participation in these trials is more burdensome both for the physicians and for the patients. It’s interesting to us that FDA is currently mandating registries for a device company seeking approval for a stent to be deployed in the renal artery. And the typical endpoint in these registries is the restenosis rate. So, a positive study would be one in which the stent was associated with a lower restenosis rate than historical controls and with an accessible complication rate. But why do we care about restenosis if opening up the artery is of no benefit in the first place? So we believe that these registries really have no place until the randomize trial is done. And if studies are not available, what about clinical practice guidelines? So this is a big trend, I think, in medicine now. If you look at the available evidence and issue these clinical practice guidelines, and I think this is a laudable effort in - on the part of physicians to try to make sense out of data and make rational decisions. But there are problems with this process. There is in fact a clinical practice guideline that’s been written for renal artery intervention that was promulgated by an august body of societies. Many of them are in interventional societies who do these procedures. And they make recommendations. Usually, they classify evidence A, B, C or D and then classify recommendations Class 1, 2 or 3. And Class 1 recommendations are recommendations for which there’s strong support in this particular clinical practice guideline. A Class 1 recommendation was issued for renal artery interventions in certain settings. And that was based on conditions for which there is evidence and/or general agreement that a given procedure is beneficial. And I would ask you, how do you reach general agreement if there’s no evidence? There is a cost to funding things based on clinical practice guidelines that they may be wrong. It contributes to a sense of complacency in the care of patients, it may increase cost without improving outcomes. And one example from my own field, in nephrology, we have clinical practice guidelines without evidence for treating bone disease and people with CKD. And in one hospital that takes care of about 500 dialysis patients, the cost of implementing these clinical practice guidelines was estimated at half million dollars per year. And again, remember there’s absolutely no evidence that this improves patient outcome. And if you extrapolate that across the whole system, it has a huge impact. There are other deleterious consequences to basing coverage on these clinical practice guidelines that are not supported by data. They serve as an impediment to performing randomized clinical trials. Will I prove something that’s already an accepted part of clinical practice? And it becomes difficult for clinicians in IRBs to deal with randomized trials if one of the groups in the trial already violates an existing clinical practice guideline even if that guideline is not well supported. And then, of course, when these things turn out to be wrong, which was the case recently in the nephrology with the erythropoietin therapy for anemia where, for many years, we’ve been treating patients through a certain hemoglobin target. Finally, two randomized trials were published and both of them showed that that was associated with worse outcomes for patients and nobody knows what to do now. And there have been probably hundreds of articles written about this conundrum since those studies were published. So, in conclusion, randomized trials are expensive, they’re difficult to perform and they’re uncommon, enrollment is a major barrier. Coverage policies, I think, do affect enrollment by altering the chances that an unproven therapy will be provided outside of the study. Registries are not the same as randomized clinical trials and can substitute for them. Clinical practice guidelines based on observational danger - data I think may increase cost without improving patient outcomes and also discourage enrollment. And from our perspective, trying to get this particular trial done, we would like to see NIH, CMS and FDA kind of work together in order to encourage the completion of these types of randomized trials which, in the final analysis, were the only way that we’ll ever really figure what’s useful and what’s not. Thank you.

Amy Abernethy: Thank you very much.